NAPIGEN is a biotechnology company focused on genome editing, specifically in mitochondrial engineering. The company develops innovative solutions aimed at enhancing food supply by creating hybrid crop plants with significantly improved yields. By producing robust male sterile lines, NAPIGEN offers advantages over traditional lines, ensuring stability and elite genetic backgrounds. This technology facilitates the development of non-GM hybrid seeds for crops that are primarily non-hybrid, such as wheat. Additionally, NAPIGEN's expertise extends to the genome engineering of various organisms, including plants, microbes, and animals, by targeting mitochondria and chloroplasts, which are essential for energy production in cells. The company's advancements are designed to meet societal needs and promote public acceptance of its biotechnological innovations.
Developer of life sciences technology intended to develop highly effective and differentiated treatments for cystic fibrosis. The company is developing first-in-class small molecules that are focused on a unique target with the potential to fully normalize CFTR function for the first time for the vast majority of people living with cystic fibrosis, enabling patients with best-in-class efficacy and reach and long-term benefit for people with cystic fibrosis.
Ansa Biotechnologies, Inc. is a biotechnology company based in Berkeley, California, focused on developing innovative DNA synthesis technology utilizing enzymes. Established in 2018, the company aims to improve upon traditional chemical methods of DNA synthesis that have remained largely unchanged for decades. Ansa's enzymatic approach offers significant advantages, including increased speed, accuracy, and cleanliness in the production of DNA fragments such as oligonucleotides. This technology enhances the capabilities of biological research, therapeutics, diagnostics, and biomanufacturing by enabling the synthesis of gene-length oligonucleotides. Through its advancements, Ansa Biotechnologies seeks to facilitate innovation in the fields of biological research and engineering, ultimately providing valuable services to medical researchers and contributing to the broader biotechnology landscape.
Synthego is a genome engineering company that enables the acceleration of life science research and development in the pursuit of improved human health.
The company leverages machine learning, automation, and gene editing to build platforms for science at scale. With its foundations in engineering disciplines, the company’s full-stack platform vertically integrates proprietary hardware, software, bioinformatics, chemistries, and molecular biology to advance both basic research and therapeutic development programs.
By providing both commercial and academic researchers and therapeutic developers with unprecedented access to cutting-edge genome engineering, Synthego is at the forefront of innovation, accelerating the development of truly engineered biology.
The company was founded in 2012 and is based in Redwood City, California.
Metagenomi, Inc. is a genetic medicines company based in Emeryville, California, focused on developing innovative gene editing systems for the treatment of genetic diseases. Since its incorporation in 2016, the company has utilized a proprietary metagenomics-derived genome editing toolbox that enables the creation of curative therapeutics. This toolbox encompasses a range of advanced technologies, including programmable nucleases, base editors, and various RNA and DNA-mediated integration systems, such as prime editing systems and CRISPR-associated transposases (CASTs). Metagenomi aims to address the challenges posed by diverse mutations that have traditionally been difficult to target with existing genome engineering approaches.
Nucleix develops, manufactures, and markets non-invasive molecular cancer diagnostic tests. Its sensitive and specific tests are based on identification of subtle changes in methylation patterns.
Nucleix offers a urine test for monitoring of bladder cancer called Bladder EpiCheck and a blood test for early detection of lung cancer called Lung EpiCheck. Adam Wasserstrom, Danny Frumkin, and Elon Ganor founded it in 2008, with its headquarters in Rehovot in Israel.
DiCE Therapeutics, founded in 2013 and based in Menlo Park, California, specializes in developing a platform for the discovery of novel small molecules aimed at targets that have previously been difficult to address in the pharmaceutical sector. The company utilizes innovative technology that extends directed evolution, allowing for the direct translation of DNA-encoded information into organic compounds, thereby streamlining the traditionally labor-intensive processes of medicinal chemistry. This platform not only serves the pharmaceutical industry but also has applications in agriculture and materials sciences. The founding team includes notable figures such as Stanford Professor Pehr Harbury, who developed the core technology, and other experienced professionals from various sectors, contributing to the company's mission of transforming drug discovery and development.
GentiBio, Inc. is a biotherapeutics company focused on developing engineered regulatory T cells (EngTregs) to treat a range of conditions, including autoimmune, alloimmune, autoinflammatory, and allergic diseases. Founded in 2020 and based in Boston, Massachusetts, with additional locations in Seattle and Israel, GentiBio utilizes a proprietary platform that combines autologous and allogeneic EngTregs. This innovative approach aims to restore immune tolerance and address the limitations found in current regulatory T-cell therapies. The company was co-founded by experts in Treg biology and synthetic immunology, emphasizing its commitment to addressing the underlying causes of diseases linked to immune system dysfunction.
PepGen is a clinical-stage biotechnology company based in Oxford, United Kingdom, focused on advancing the delivery of nucleic acid therapeutics, particularly antisense oligonucleotides. Established in 2018, PepGen aims to enhance the clinical efficacy of these therapeutics through its proprietary Enhanced Delivery Oligonucleotide (EDO) platform. This innovative technology employs engineered peptides designed for optimized tissue penetration, cellular uptake, and nuclear delivery, enabling effective transport of oligonucleotides to various target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system. By leveraging its drug delivery capabilities, PepGen seeks to transform the treatment landscape for severe neuromuscular and neurological diseases.
Element Biosciences, Inc. develops genetic analysis tools for the research and diagnostic markets. The company provides a disruptive DNA sequencing technology and a sequencing platform, such as surface chemistry, sequencing chemistry, detection, and data analysis. The company was incorporated in 2017 and is based in San Diego, California.
Affinia Therapeutics Inc. is a biotechnology company focused on developing gene therapies for various diseases, particularly those affecting muscle and the central nervous system (CNS). Founded in 2019 and based in Waltham, Massachusetts, the company specializes in creating rationally designed adeno-associated virus (AAV) vectors. By integrating synthetic and systems biology with high-throughput screening and advanced resolution techniques, Affinia Therapeutics aims to address significant unmet medical needs. The company’s innovative approach allows for the development of novel gene therapies with enhanced properties, targeting the treatment of devastating diseases and improving the lives of affected individuals.
Forge Biologics, Inc. is a contract development and manufacturing organization based in Columbus, Ohio, focused on developing and manufacturing gene therapies and genetic medicines. The company specializes in viral vector gene therapies aimed at treating genetic diseases, including a novel approach combining adeno-associated virus (AAV) with umbilical cord transplants for infantile Krabbe disease, a severe neurodegenerative condition. Founded in 2019, Forge Biologics partners with scientists, healthcare professionals, and biotech and pharmaceutical companies to facilitate the transition of gene therapy programs from preclinical development to clinical and commercial-scale production. By providing end-to-end capabilities, the company aims to accelerate the availability of transformative medicines for patients suffering from rare genetic disorders.
Nucleix develops, manufactures, and markets non-invasive molecular cancer diagnostic tests. Its sensitive and specific tests are based on identification of subtle changes in methylation patterns.
Nucleix offers a urine test for monitoring of bladder cancer called Bladder EpiCheck and a blood test for early detection of lung cancer called Lung EpiCheck. Adam Wasserstrom, Danny Frumkin, and Elon Ganor founded it in 2008, with its headquarters in Rehovot in Israel.
Scribe Therapeutics Inc. is a biotechnology company focused on developing innovative therapeutics using CRISPR technology to address genetic disorders. Founded in 2017 and based in Berkeley, California, Scribe specializes in engineering CRISPR enzymes known as X-Editing (XE) molecules, which enhance the efficacy, specificity, and deliverability of genome editing compared to existing methods. The company's proprietary platform enables the creation of custom-engineered enzymes and delivery systems, facilitating precision targeting within the genome. Scribe Therapeutics aims to establish CRISPR-based therapies as a standard in clinical care, expanding access to transformative treatments for a wide range of conditions, including immuno-oncology and degenerative disorders. Through its commitment to overcoming the limitations of current genome editing technologies, Scribe Therapeutics is poised to make substantial contributions to human therapeutics.
Graphite Bio is a gene editing company focused on targeted DNA integration to address severe diseases. The company employs technology that enables the insertion of new genetic sequences to replace defective genes, repair damaged portions of genes, and insert therapeutic genetic material at specific locations within the genome. This precise approach leverages natural cellular DNA repair mechanisms to enhance the efficiency of gene integration, aiming to correct the underlying causes of various diseases. Founded in 2019 and based in South San Francisco, California, Graphite Bio was previously known as Integral Medicines, Inc. and rebranded in August 2020. The company is committed to advancing gene replacement therapies and potential cures for individuals affected by serious health conditions.
Orchard Therapeutics is a biopharmaceutical company based in London, focused on developing innovative gene therapies for serious and life-threatening rare diseases. Operating in the United Kingdom, European Union, and the United States, Orchard employs an autologous ex vivo gene therapy approach to transform patients’ hematopoietic stem cells into gene-modified products for treatment through a single administration. Its portfolio includes Strimvelis, the first approved gene therapy for adenosine deaminase-severe combined immunodeficiency (ADA-SCID), along with several products in clinical development targeting metachromatic leukodystrophy, Wiskott-Aldrich syndrome, X-linked chronic granulomatous disease, and transfusion-dependent beta-thalassemia. The company also has a robust preclinical pipeline addressing various mucopolysaccharidosis types. Orchard collaborates with leading institutions in gene therapy, enhancing its research capabilities and clinical programs. Founded in 2015, Orchard Therapeutics continues to demonstrate a commitment to transforming the lives of patients through its advanced therapies.
Design Therapeutics, Inc. is a clinical-stage biotechnology company based in Solana Beach, California, focused on developing innovative therapies for degenerative disorders resulting from nucleotide repeat expansions. Founded in 2017, the company is advancing its lead program aimed at treating Friedreich's ataxia, while also exploring treatment options for other conditions such as Fragile X syndrome and myotonic dystrophy. Design Therapeutics is known for its GeneTACTM molecules, a new class of small-molecule gene-targeted therapies designed to address the underlying causes of diseases linked to inherited nucleotide repeat expansion mutations. The company's ongoing research efforts seek to develop disease-modifying treatments for serious conditions driven by these genetic factors.
Verve Therapeutics is a biotechnology company focused on developing innovative therapies aimed at editing the adult human genome to provide lifelong protection against coronary artery disease, a leading global cause of mortality. Founded in 2018 and based in Cambridge, Massachusetts, with a research facility in Philadelphia, the company employs gene-editing technology to create treatments that shift the management of cardiovascular diseases from chronic care to potentially curative interventions. Its initial programs concentrate on the PCSK9 and ANGPTL3 genes, which are recognized for their roles in regulating blood lipids, specifically low-density lipoprotein cholesterol. Verve Therapeutics collaborates with partners such as Beam Therapeutics and Verily to enhance delivery technologies for its cardiovascular treatments.
DiCE Therapeutics, founded in 2013 and based in Menlo Park, California, specializes in developing a platform for the discovery of novel small molecules aimed at targets that have previously been difficult to address in the pharmaceutical sector. The company utilizes innovative technology that extends directed evolution, allowing for the direct translation of DNA-encoded information into organic compounds, thereby streamlining the traditionally labor-intensive processes of medicinal chemistry. This platform not only serves the pharmaceutical industry but also has applications in agriculture and materials sciences. The founding team includes notable figures such as Stanford Professor Pehr Harbury, who developed the core technology, and other experienced professionals from various sectors, contributing to the company's mission of transforming drug discovery and development.
Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic disorder primarily affecting boys. The company's lead candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in muscle tissues. In addition to SGT-001, Solid Biosciences is advancing Anti-LTBP4, a monoclonal antibody designed to mitigate fibrosis and inflammation by targeting the LTBP4 protein. The company also works on developing biomarkers and wearable assistive devices under the Solid Suit program, which aims to provide therapeutic benefits to patients. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to further develop and commercialize gene therapies for DMD. The company’s initiatives address a critical need, as there are currently no effective cures or satisfactory treatments for this progressive and life-threatening condition.
PepGen is a clinical-stage biotechnology company based in Oxford, United Kingdom, focused on advancing the delivery of nucleic acid therapeutics, particularly antisense oligonucleotides. Established in 2018, PepGen aims to enhance the clinical efficacy of these therapeutics through its proprietary Enhanced Delivery Oligonucleotide (EDO) platform. This innovative technology employs engineered peptides designed for optimized tissue penetration, cellular uptake, and nuclear delivery, enabling effective transport of oligonucleotides to various target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system. By leveraging its drug delivery capabilities, PepGen seeks to transform the treatment landscape for severe neuromuscular and neurological diseases.
PepGen is a clinical-stage biotechnology company based in Oxford, United Kingdom, focused on advancing the delivery of nucleic acid therapeutics, particularly antisense oligonucleotides. Established in 2018, PepGen aims to enhance the clinical efficacy of these therapeutics through its proprietary Enhanced Delivery Oligonucleotide (EDO) platform. This innovative technology employs engineered peptides designed for optimized tissue penetration, cellular uptake, and nuclear delivery, enabling effective transport of oligonucleotides to various target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system. By leveraging its drug delivery capabilities, PepGen seeks to transform the treatment landscape for severe neuromuscular and neurological diseases.
Metagenomi, Inc. is a genetic medicines company based in Emeryville, California, focused on developing innovative gene editing systems for the treatment of genetic diseases. Since its incorporation in 2016, the company has utilized a proprietary metagenomics-derived genome editing toolbox that enables the creation of curative therapeutics. This toolbox encompasses a range of advanced technologies, including programmable nucleases, base editors, and various RNA and DNA-mediated integration systems, such as prime editing systems and CRISPR-associated transposases (CASTs). Metagenomi aims to address the challenges posed by diverse mutations that have traditionally been difficult to target with existing genome engineering approaches.
AavantiBio, Inc. is dedicated to developing gene transfer therapy and gene editing technologies aimed at improving the lives of individuals afflicted by rare and fatal diseases. The company primarily focuses on Friedreich’s Ataxia, a rare inherited genetic disorder that leads to significant cardiac and central nervous system dysfunction. Founded in 2019 and based in Gainesville, Florida, AavantiBio is committed to advancing innovative gene therapies to address unmet medical needs in the treatment of rare genetic diseases and autoimmune conditions. Through its research and development efforts, the company seeks to transform patient care and enhance treatment options for those affected by these serious health challenges.
Kinnate Biopharma develops precision oncology therapeutics to treat patients with genomically-defined cancers. Its mission is to expand the reach of targeted therapeutics by developing products for underserved populations.
The company utilizes its deep expertise in structure-based drug discovery, translational research, and patient-driven precision medicine. Its programs include candidates in preclinical development for cancers that are driven by specific oncogenic alterations in either the BRAF kinase gene or in the FGFR2 and FGFR3 kinase genes.
Synthego is a genome engineering company that enables the acceleration of life science research and development in the pursuit of improved human health.
The company leverages machine learning, automation, and gene editing to build platforms for science at scale. With its foundations in engineering disciplines, the company’s full-stack platform vertically integrates proprietary hardware, software, bioinformatics, chemistries, and molecular biology to advance both basic research and therapeutic development programs.
By providing both commercial and academic researchers and therapeutic developers with unprecedented access to cutting-edge genome engineering, Synthego is at the forefront of innovation, accelerating the development of truly engineered biology.
The company was founded in 2012 and is based in Redwood City, California.
GentiBio, Inc. is a biotherapeutics company focused on developing engineered regulatory T cells (EngTregs) to treat a range of conditions, including autoimmune, alloimmune, autoinflammatory, and allergic diseases. Founded in 2020 and based in Boston, Massachusetts, with additional locations in Seattle and Israel, GentiBio utilizes a proprietary platform that combines autologous and allogeneic EngTregs. This innovative approach aims to restore immune tolerance and address the limitations found in current regulatory T-cell therapies. The company was co-founded by experts in Treg biology and synthetic immunology, emphasizing its commitment to addressing the underlying causes of diseases linked to immune system dysfunction.
Element Biosciences, Inc. develops genetic analysis tools for the research and diagnostic markets. The company provides a disruptive DNA sequencing technology and a sequencing platform, such as surface chemistry, sequencing chemistry, detection, and data analysis. The company was incorporated in 2017 and is based in San Diego, California.
Prevail Therapeutics Inc. is a biotechnology company based in New York that specializes in developing gene therapies for neurodegenerative diseases. Founded in 2017 with support from The Silverstein Foundation for Parkinson’s with GBA, REGENXBIO, and OrbiMed, the company is focused on creating innovative adeno-associated virus (AAV)-based treatments. Its lead candidate, PR001, is currently undergoing Phase I/II clinical trials aimed at treating Parkinson's disease linked to GBA1 mutations and neuronopathic Gaucher disease. Additionally, Prevail is developing PR006 for frontotemporal dementia associated with GRN mutations and PR004 for various synucleinopathies. The company is dedicated to advancing therapies that can modify the course of these debilitating conditions.
Ervaxx is pioneering the use of Dark Antigens™ to deliver targeted off-the-shelf cancer vaccines and other immunotherapies for treating and preventing cancer. Their Dark Antigens™ derive from vast untapped expanses of genetic ‘dark matter’ beyond the normal coding regions of the genome, which are generally silenced in normal tissue but are selectively activated in cancer.
Stoke Therapeutics, Inc., an early-stage biopharmaceutical company, develops novel antisense oligonucleotide medicines to treat the underlying causes of severe genetic diseases. Its lead product candidate, STK-001 used to treat Dravet syndrome, a severe and progressive genetic epilepsy. Stoke Therapeutics, Inc. has a partnership with Invitae Corporation to offer genetic testing. The company was formerly known as ASOthera Pharmaceuticals, Inc. and changed its name to Stoke Therapeutics, Inc. in May 2016. Stoke Therapeutics, Inc. was founded in 2014 and is headquartered in Bedford, Massachusetts.
Orchard Therapeutics is a biopharmaceutical company based in London, focused on developing innovative gene therapies for serious and life-threatening rare diseases. Operating in the United Kingdom, European Union, and the United States, Orchard employs an autologous ex vivo gene therapy approach to transform patients’ hematopoietic stem cells into gene-modified products for treatment through a single administration. Its portfolio includes Strimvelis, the first approved gene therapy for adenosine deaminase-severe combined immunodeficiency (ADA-SCID), along with several products in clinical development targeting metachromatic leukodystrophy, Wiskott-Aldrich syndrome, X-linked chronic granulomatous disease, and transfusion-dependent beta-thalassemia. The company also has a robust preclinical pipeline addressing various mucopolysaccharidosis types. Orchard collaborates with leading institutions in gene therapy, enhancing its research capabilities and clinical programs. Founded in 2015, Orchard Therapeutics continues to demonstrate a commitment to transforming the lives of patients through its advanced therapies.
Precision BioSciences, Inc. is a biotechnology company that specializes in genome editing, utilizing its proprietary ARCUS platform to develop therapeutic and food products. The Therapeutic segment focuses on creating allogeneic CAR T immunotherapies aimed at treating various cancers, including acute lymphoblastic leukemia and multiple myeloma, with several candidates currently in clinical trials. Notable product candidates include PBCAR0191, targeting CD19, and PBCAR269A, targeting BCMA. Additionally, the company is engaged in in vivo gene correction and has collaborations for developing treatments for chronic Hepatitis B. The Food segment aims to innovate in food and nutrition products. Founded in 2006 and headquartered in Durham, North Carolina, Precision BioSciences is committed to translating advanced genome editing technology into impactful medical and agricultural solutions.
Prevail Therapeutics Inc. is a biotechnology company based in New York that specializes in developing gene therapies for neurodegenerative diseases. Founded in 2017 with support from The Silverstein Foundation for Parkinson’s with GBA, REGENXBIO, and OrbiMed, the company is focused on creating innovative adeno-associated virus (AAV)-based treatments. Its lead candidate, PR001, is currently undergoing Phase I/II clinical trials aimed at treating Parkinson's disease linked to GBA1 mutations and neuronopathic Gaucher disease. Additionally, Prevail is developing PR006 for frontotemporal dementia associated with GRN mutations and PR004 for various synucleinopathies. The company is dedicated to advancing therapies that can modify the course of these debilitating conditions.
Dyne Therapeutics, Inc., a muscle disease company, operates as a biotechnology company that focuses on advancing therapeutics for genetically driven muscle diseases in the United States. It develops various programs for myotonic dystrophy type 1, duchenne muscular dystrophy, and facioscapulohumeral dystrophy, as well as rare skeletal muscle, and cardiac and metabolic muscle diseases using its FORCE platform that delivers disease-modifying therapies. The company was founded in 2017 and is based in Waltham, Massachusetts.
Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic disorder primarily affecting boys. The company's lead candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in muscle tissues. In addition to SGT-001, Solid Biosciences is advancing Anti-LTBP4, a monoclonal antibody designed to mitigate fibrosis and inflammation by targeting the LTBP4 protein. The company also works on developing biomarkers and wearable assistive devices under the Solid Suit program, which aims to provide therapeutic benefits to patients. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to further develop and commercialize gene therapies for DMD. The company’s initiatives address a critical need, as there are currently no effective cures or satisfactory treatments for this progressive and life-threatening condition.
Dicerna Pharmaceuticals is a biopharmaceutical company dedicated to the discovery and development of innovative treatments primarily for liver-related diseases, including rare inherited conditions, chronic liver diseases, and viral infections. Utilizing its proprietary GalXC RNA interference technology, Dicerna focuses on silencing disease-causing genes to address various health issues. Key product candidates include nedosiran for primary hyperoxaluria, RG6346 for chronic hepatitis B virus infection, and belcesiran for genetic liver disease, among others. The company's extensive research efforts also encompass treatments for cardiovascular diseases and neurodegeneration. Dicerna has established strategic collaborations with prominent pharmaceutical companies to enhance its development capabilities. Founded in 2006 and headquartered in Lexington, Massachusetts, Dicerna is at the forefront of advancing RNA-based therapeutics.
Audentes Therapeutics is a clinical-stage biotechnology company dedicated to developing and commercializing gene therapy products for patients with serious, life-threatening rare diseases caused by single gene defects. The company is advancing several therapies, including AT132, currently in Phase I/II clinical trials for X-linked myotubular myopathy, and AT342, also in Phase I/II studies for Crigler-Najjar syndrome. Additionally, Audentes is exploring AT845 in preclinical studies for Pompe disease and AT307 for the CASQ2 subtype of catecholaminergic polymorphic ventricular tachycardia. The company is also working on vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. Collaborations include a partnership with Genethon for XLMTM treatments and an agreement with the University of Pennsylvania for Crigler-Najjar syndrome products. Founded in 2012 and headquartered in San Francisco, California, Audentes Therapeutics operates as a subsidiary of Astellas US Holding, Inc.
Based in Dallas, Texas, AveXis is a clinic-ready, synthetic biology platform company. AveXis has, at its core, a desire to establish unique industry and research alliances which will bring innovative treatments to people with unmet medical needs. Our work in spinal muscular atrophy (SMA), a rare/orphan disease, is our first focus.
WaVe Life Sciences Ltd. is a clinical stage genetic medicine company focused on developing novel stereopure oligonucleotides through its proprietary PRISM platform. The company aims to address genetic defects by either reducing the expression of harmful proteins or converting dysfunctional mutant proteins into functional ones. It primarily concentrates on neurological disorders affecting the central and neuromuscular systems. WaVe Life Sciences has established research and collaboration agreements with major pharmaceutical companies to advance its oligonucleotide therapeutics. Founded in 2012, the company is headquartered in Singapore and maintains research facilities in Boston and Japan. The organization was established by leading scientists in the field of chemistry and life sciences, underscoring its commitment to innovative approaches in nucleic acid therapeutics.
Precision BioSciences, Inc. is a biotechnology company that specializes in genome editing, utilizing its proprietary ARCUS platform to develop therapeutic and food products. The Therapeutic segment focuses on creating allogeneic CAR T immunotherapies aimed at treating various cancers, including acute lymphoblastic leukemia and multiple myeloma, with several candidates currently in clinical trials. Notable product candidates include PBCAR0191, targeting CD19, and PBCAR269A, targeting BCMA. Additionally, the company is engaged in in vivo gene correction and has collaborations for developing treatments for chronic Hepatitis B. The Food segment aims to innovate in food and nutrition products. Founded in 2006 and headquartered in Durham, North Carolina, Precision BioSciences is committed to translating advanced genome editing technology into impactful medical and agricultural solutions.
Dimension Therapeutics, Inc. is a gene therapy company dedicated to developing innovative treatments for rare and metabolic diseases linked to liver function. Founded in 2013 and based in Cambridge, Massachusetts, the company has established a robust pipeline of gene therapy candidates. Its lead products include DTX101 for hemophilia B and DTX201 for hemophilia A, alongside other programs targeting conditions such as ornithine transcarbamylase deficiency, glycogen storage disease type Ia, phenylketonuria, Wilson disease, and citrullinemia type I. Dimension Therapeutics collaborates with REGENX Biosciences, leveraging its extensive patent portfolio related to NAV vector technology. This partnership enhances Dimension's ability to advance its gene therapy initiatives, particularly in the realm of adeno-associated virus therapeutics. In 2017, Dimension Therapeutics became a subsidiary of Ultragenyx Pharmaceutical Inc.
Natera is a diagnostic and research company specializing in genetic testing services, primarily focused on preconception and prenatal testing. Its flagship product, Panorama, is a non-invasive prenatal test that screens for chromosomal abnormalities in fetuses through a blood draw from the mother, including assessments for twin pregnancies. Other offerings include Vistara, which tests for single-gene disorders, and Horizon carrier screening to identify carrier status for various genetic diseases. Natera also provides the Anora test for analyzing fetal chromosomes in cases of miscarriage and non-invasive paternity testing. Additionally, the company has developed Signatera, a technology that detects circulating tumor DNA to monitor cancer recurrence. Natera utilizes a direct sales force and a network of approximately 100 laboratory and distribution partners for product distribution. The company was founded in 2003, has undergone several name changes, and is headquartered in San Carlos, California. Its collaborations with organizations like BGI Genomics and Foundation Medicine further enhance its capabilities in genetic testing and personalized medicine.
Aeglea BioTherapeutics is a clinical-stage biotechnology company focused on developing human enzyme therapeutics for rare genetic and cancer diseases. Its lead product candidate, pegzilarginase, is a recombinant human Arginase 1 enzyme currently undergoing Phase III trials to assess its safety and efficacy in treating Arginase 1 deficiency. The company's preclinical pipeline includes several innovative candidates: ACN00177 for homocystinuria, AEB5100 which degrades plasma cystine and cysteine, AEB2109 targeting the degradation of methionine, and AEB3103. Aeglea BioTherapeutics aims to exploit the amino acid dependencies of tumors by creating novel, human-derived enzymes that selectively target these vulnerabilities, differentiating the metabolic needs of tumor cells from those of normal tissues. Founded in 2013 and headquartered in Austin, Texas, the company was formerly known as Aeglea BioTherapeutics Holdings, LLC before changing its name in 2015.
Wave Life Sciences
Series A in 2015
Wave Life Sciences (Nasdaq: WVE) is a clinical-stage RNA medicines company committed to delivering life-changing treatments for people battling devastating diseases. Wave aspires to develop best-in-class medicines across multiple therapeutic modalities using PRISM, the company’s proprietary discovery and drug development platform that enables the precise design, optimization, and production of stereopure oligonucleotides. Driven by a resolute sense of urgency, the Wave team is targeting a broad range of genetically defined diseases so that patients and families may realize a brighter future. To find out more, please visit www.wavelifesciences.com and follow Wave on Twitter @WaveLifeSci.
Dicerna Pharmaceuticals is a biopharmaceutical company dedicated to the discovery and development of innovative treatments primarily for liver-related diseases, including rare inherited conditions, chronic liver diseases, and viral infections. Utilizing its proprietary GalXC RNA interference technology, Dicerna focuses on silencing disease-causing genes to address various health issues. Key product candidates include nedosiran for primary hyperoxaluria, RG6346 for chronic hepatitis B virus infection, and belcesiran for genetic liver disease, among others. The company's extensive research efforts also encompass treatments for cardiovascular diseases and neurodegeneration. Dicerna has established strategic collaborations with prominent pharmaceutical companies to enhance its development capabilities. Founded in 2006 and headquartered in Lexington, Massachusetts, Dicerna is at the forefront of advancing RNA-based therapeutics.
Bluebird Bio is a clinical-stage biotechnology company focused on developing transformative gene therapies for severe genetic diseases and cancer. The company is engaged in researching and commercializing therapies that aim to genetically modify patients' cells to correct the underlying genetic causes of diseases. Key programs include LentiGlobin for β-thalassemia and sickle cell disease, and Lenti-D for cerebral adrenoleukodystrophy. In oncology, bluebird bio is developing CAR T cell therapies, such as bb2121 and bb21217, for multiple myeloma. The company has formed strategic collaborations with Bristol-Myers Squibb and Regeneron Pharmaceuticals to advance gene therapies in oncology, and works with various partners to develop innovative solutions in cancer treatment and in vivo genome editing for genetic disorders. Founded in 1992 and headquartered in Cambridge, Massachusetts, bluebird bio generates revenue through collaboration arrangements, research fees, license fees, and grants.
CellScape Corporation (formerly Saryna Medical Corp) is a privately funded company headquartered in Newark, California, in the heart of the biotech corridor of the San Francisco Bay Area. The Company has developed whole fetal cell technology to transform prenatal genetic testing using a simple blood test. Their vision is to make comprehensive prenatal genetic testing available to every woman without requiring an invasive procedure that might put the fetus at risk. Their leadership team is accomplished in bringing innovative, cost-effective medical and molecular diagnostics to market.
OvaScience, Inc. was a fertility company dedicated to developing and commercializing innovative treatment options for women facing infertility challenges. The company focused on its patented technology involving the discovery of egg precursor (EggPC) cells, which aimed to transform fertility treatments. Key offerings included OvaPrime, which sought to restore egg production by utilizing a woman’s own EggPC cells; OvaTure, designed to mature these cells into fertilizable eggs outside the body; and AUGMENT, which enhanced fertilization and pregnancy rates by incorporating mitochondria from a woman’s EggPC cells. Founded in 2011 and headquartered in Waltham, Massachusetts, OvaScience was co-founded by notable figures from the life sciences field and was backed by prominent investors. In December 2018, OvaScience was acquired by Millendo Therapeutics in a reverse merger transaction.
Eiger BioPharmaceuticals is a late-stage biopharmaceutical company based in Palo Alto, California, dedicated to developing targeted therapies for rare and ultra-rare diseases. The company’s lead product candidate, Lonafarnib, is an orally bioavailable small molecule currently undergoing Phase 3 clinical trials for the treatment of hepatitis delta virus (HDV) infection. Eiger's pipeline also includes Lambda, which is in Phase 2 trials and targets type III interferon receptors for HDV, as well as Lonafarnib for progeria and progeroid laminopathies. Additionally, the company is developing Avexitide, which has completed Phase 2 trials for post-bariatric hypoglycemia and is also in Phase 2 for congenital hyperinsulinism. Eiger's focus is on addressing high unmet medical needs in serious diseases where no approved therapies currently exist.
Genelabs Technologies Inc., founded in 1983 and headquartered in Redwood City, California, is a biopharmaceutical company focused on the discovery and development of therapies for infectious diseases. The company has a product pipeline that includes projects targeting hepatitis C virus infection, as well as late-stage clinical assets such as an investigational vaccine for hepatitis E virus, which is being developed in collaboration with GlaxoSmithKline. Another notable asset is Prestara, an investigational drug for systemic lupus erythematosus. Genelabs Technologies has established collaborative agreements with the Novartis Institutes for BioMedical Research to advance research on hepatitis C virus polymerase and with the National Health Research Institutes and Genovate Biotechnology Co., Ltd. to develop compounds targeting the hepatitis C virus. In 2009, Genelabs Technologies was acquired by SmithKline Beecham Corporation.